EP 3863677 A4 20221228 - POLY(Beta-AMINO ESTER) NANOPARTICLES FOR THE NON-VIRAL DELIVERY OF PLASMID DNA FOR GENE EDITING AND RETINAL GENE THERAPY
Title (en)
POLY(Beta-AMINO ESTER) NANOPARTICLES FOR THE NON-VIRAL DELIVERY OF PLASMID DNA FOR GENE EDITING AND RETINAL GENE THERAPY
Title (de)
POLY(BETA-AMINO-ESTER)-NANOPARTIKEL ZUR NICHT-VIRALEN ABGABE VON PLASMID-DNA FÜR DIE GENBEARBEITUNG UND DIE RETINALE GENTHERAPIE
Title (fr)
NANOPARTICULES DE POLY (Beta-AMINO ESTER) POUR L'ADMINISTRATION NON VIRALE D'ADN DE PLASMIDE POUR L'ÉDITION GÉNIQUE ET LA THÉRAPIE GÉNIQUE DE RÉTINE
Publication
Application
Priority
- US 201862743883 P 20181010
- US 2019055741 W 20191010
Abstract (en)
[origin: WO2020077159A1] Biodegradable particles for delivering a nucleic acid encoding gene-editing factors or a nucleic acid associated with a therapeutic protein to a cell, and compositions, methods, systems, and kits for gene editing in vivo or ex vivo or gene therapy for treating retinal eye diseases are disclosed.
IPC 8 full level
A61K 47/34 (2017.01); A61K 9/51 (2006.01); A61K 47/14 (2017.01); A61K 48/00 (2006.01); C08G 69/26 (2006.01)
CPC (source: EP US)
C08G 69/26 (2013.01 - EP US); C12N 15/111 (2013.01 - EP US); C12N 15/85 (2013.01 - EP); C12N 15/88 (2013.01 - EP US); C12N 15/907 (2013.01 - EP); A61K 9/51 (2013.01 - EP); A61K 48/00 (2013.01 - EP); C12N 2310/20 (2017.04 - EP); C12N 2320/32 (2013.01 - EP)
Citation (search report)
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- [XDI] DEZHONG ZHOU ET AL: "Poly(beta-Amino Ester)-Nanoparticle Mediated Transfection of Retinal Pigment Epithelial Cells In Vitro and In Vivo", SCIENCE ADVANCES, vol. 2, no. 6, 1 June 2016 (2016-06-01), pages e1600102, XP055539263, DOI: 10.1126/sciadv.1600102
- [XDI] ZHU DA ET AL: "Nanoparticles Based on Poly ([beta]-Amino Ester) and HPV16-Targeting CRISPR/shRNA as Potential Drugs for HPV16-Related Cervical Malignancy", MOLECULAR THERAPY, vol. 26, no. 10, 1 October 2018 (2018-10-01), US, pages 2443 - 2455, XP055907040, ISSN: 1525-0016, Retrieved from the Internet <URL:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6171328/pdf/main.pdf> DOI: 10.1016/j.ymthe.2018.07.019
- [XI] JAMES CLIFF KACZMAREK: "Development of Polymer -Lipid Nanoparticles for Potent mRNA Delivery to the Lung", 30 September 2018 (2018-09-30), XP055682159, Retrieved from the Internet <URL:https://dspace.mit.edu/bitstream/handle/1721.1/121895/1103314257-MIT.pdf?sequence=1&isAllowed=y> [retrieved on 20200402]
- [XP] RUI YUAN ET AL: "Reducible Branched Ester-Amine Quadpolymers (rBEAQs) Codelivering Plasmid DNA and RNA Oligonucleotides Enable CRISPR/Cas9 Genome Editing", APPLIED MATERIALS & INTERFACES, vol. 11, no. 11, 22 February 2019 (2019-02-22), US, pages 10472 - 10480, XP055925498, ISSN: 1944-8244, DOI: 10.1021/acsami.8b20206
- [XP] WILSON DAVID R. ET AL: "Differentially Branched Ester Amine Quadpolymers with Amphiphilic and pH-Sensitive Properties for Efficient Plasmid DNA Delivery", MOLECULAR PHARMACEUTICS, vol. 16, no. 2, 4 February 2019 (2019-02-04), US, pages 655 - 668, XP055840765, ISSN: 1543-8384, Retrieved from the Internet <URL:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7297465/pdf/nihms-1581884.pdf> DOI: 10.1021/acs.molpharmaceut.8b00963
- [XI] KOZIELSKI KRISTEN L. ET AL: "Bioreducible Cationic Polymer-Based Nanoparticles for Efficient and Environmentally Triggered Cytoplasmic siRNA Delivery to Primary Human Brain Cancer Cells", ACS NANO, vol. 8, no. 4, 27 March 2014 (2014-03-27), US, pages 3232 - 3241, XP055958014, ISSN: 1936-0851, DOI: 10.1021/nn500704t
- [XI] KRISTEN L. KOZIELSKI ET AL: "A bioreducible linear poly(β-amino ester) for siRNA delivery", CHEMICAL COMMUNICATIONS, vol. 49, no. 46, 1 January 2013 (2013-01-01), UK, pages 5319, XP055341016, ISSN: 1359-7345, DOI: 10.1039/c3cc40718g
- [XI] CUTLAR LARA ET AL: "Highly Branched Poly([beta]-Amino Esters): Synthesis and Application in Gene Delivery", BIOMACROMOLECULES, vol. 16, no. 9, 25 August 2015 (2015-08-25), US, pages 2609 - 2617, XP055921486, ISSN: 1525-7797, Retrieved from the Internet <URL:https://pubs.acs.org/doi/pdf/10.1021/acs.biomac.5b00966> DOI: 10.1021/acs.biomac.5b00966
- [XI] LIU SHUAI ET AL: "Biodegradable Highly Branched Poly([beta]-Amino Ester)s for Targeted Cancer Cell Gene Transfection", ACS BIOMATERIALS SCIENCE & ENGINEERING, vol. 3, no. 7, 26 October 2016 (2016-10-26), pages 1283 - 1286, XP055925529, ISSN: 2373-9878, DOI: 10.1021/acsbiomaterials.6b00503
- [XI] YONGSHENG GAO ET AL: "Highly Branched Poly(?-amino esters) for Non-Viral Gene Delivery: High Transfection Efficiency and Low Toxicity Achieved by Increasing Molecular Weight", BIOMACROMOLECULES, vol. 17, no. 11, 3 October 2016 (2016-10-03), US, pages 3640 - 3647, XP055539247, ISSN: 1525-7797, DOI: 10.1021/acs.biomac.6b01120
- [XI] ZHOU DEZHONG ET AL: "Highly branched poly([beta]-amino ester)s for skin gene therapy", JOURNAL OF CONTROLLED RELEASE, ELSEVIER, AMSTERDAM, NL, vol. 244, 8 June 2016 (2016-06-08), pages 336 - 346, XP029850289, ISSN: 0168-3659, DOI: 10.1016/J.JCONREL.2016.06.014
- [XI] SHUAI LIU ET AL: "Alkylated branched poly(beta-amino esters) demonstrate strong DNA encapsulation, high nanoparticle stability and robust gene transfection efficacy", JOURNAL OF MATERIALS CHEMISTRY. B, vol. 5, no. 27, 1 January 2017 (2017-01-01), GB, pages 5307 - 5310, XP055539258, ISSN: 2050-750X, DOI: 10.1039/C7TB00996H & LIU SHUAI ET AL: "Supplementary information.- Alkylated branched poly([beta]-amino esters) demonstrate strong DNA encapsulation, high nanoparticle stability and robust gene transfection efficacy", JOURNAL OF MATERIALS CHEMISTRY. B, vol. 5, no. 27, 1 January 2017 (2017-01-01), GB, pages 5307 - 5310, XP055971350, ISSN: 2050-750X, DOI: 10.1039/C7TB00996H
- [XI] JIAN-YUAN HUANG ET AL: "Tailoring highly branched poly(β-amino ester)s: a synthetic platform for epidermal gene therapy", CHEMICAL COMMUNICATIONS, vol. 51, no. 40, 14 April 2015 (2015-04-14), pages 8473 - 8476, XP055211464, ISSN: 1359-7345, DOI: 10.1039/C5CC02193F & HUANG JIAN-YUAN ET AL: "Supplementary information.- Tailoring highly branched poly([beta]-amino ester)s: a synthetic platform for epidermal gene therapy", CHEMICAL COMMUNICATIONS, vol. 51, no. 40, 1 January 2015 (2015-01-01), UK, pages 8473 - 8476, XP055971365, ISSN: 1359-7345, DOI: 10.1039/C5CC02193F
- See references of WO 2020077159A1
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
DOCDB simple family (publication)
WO 2020077159 A1 20200416; CN 113194998 A 20210730; EP 3863677 A1 20210818; EP 3863677 A4 20221228; US 2023002790 A1 20230105
DOCDB simple family (application)
US 2019055741 W 20191010; CN 201980082833 A 20191010; EP 19870203 A 20191010; US 201917282939 A 20191010